Spinal Muscular Atrophy Press Releases

Stem cells have been hailed as a toolkit to treat a host of diseases, but at an NIH symposium on May 6, researchers said they are still deciphering the toolkit’s instruction manual.

In multiple sclerosis (MS), the immune cells that patrol our blood for pathogens venture out of the bloodstream and attack the brain. Researchers have found that leakage of a blood-clotting protein into the brain, once considered merely a sign of damage in the MS brain, helps stimulate this attack.

Drug therapy can extend survival and improve movement in a mouse model of spinal muscular atrophy (SMA), new research shows. The study, carried out at the NIH’s National Institute of Neurological Disorders and Stroke (NINDS), suggests that similar drugs might one day be useful for treating human SMA.

Drug therapy can extend survival and improve movement in a mouse model of spinal muscular atrophy (SMA), new research shows. The study, carried out at the NIH’s National Institute of Neurological Disorders and Stroke (NINDS), suggests that similar drugs might one day be useful for treating human SMA.

For the first time, researchers have enticed transplants of embryonic stem cell-derived motor neurons in the spinal cord to connect with muscles and partially restore function in paralyzed animals. The study suggests that similar techniques may be useful for treating such disorders as spinal cord injury, transverse myelitis, amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy. The study was funded in part by the NIH’s National Institute of Neurological Disorders and Stroke (NINDS).

For the first time, researchers have enticed transplants of embryonic stem cell-derived motor neurons in the spinal cord to connect with muscles and partially restore function in paralyzed animals. The study suggests that similar techniques may be useful for treating such disorders as spinal cord injury, transverse myelitis, amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy. The study was funded in part by the NIH’s National Institute of Neurological Disorders and Stroke (NINDS).

New research suggests that an off-the-market pain reliever called indoprofen may be a starting point for finding a new drug to treat spinal muscular atrophy (SMA), a devastating childhood neurological disorder.

New research suggests that an off-the-market pain reliever called indoprofen may be a starting point for finding a new drug to treat spinal muscular atrophy (SMA), a devastating childhood neurological disorder.

Many neurological diseases occur when specific groups of neurons die because of nerve damage, toxins, inflammation, or other factors. A new study suggests that activity of a single gene can stop neurons from dying regardless of what triggers this process. The findings could lead to new ways of treating neurodegenerative diseases.
Fact Sheet

One of the first studies of valproic acid as a potential therapy for spinal muscular atrophy (SMA) shows that, in cultured cells, the drug increases production of a protein that is reduced or missing in people with the disorder. While preliminary, the study suggests that valproic acid or related drugs may be able to halt or even reverse the course of this devastating childhood disease.
Fact Sheet

Many neurological diseases occur when specific groups of neurons die because of nerve damage, toxins, inflammation, or other factors. A new study suggests that activity of a single gene can stop neurons from dying regardless of what triggers this process. The findings could lead to new ways of treating neurodegenerative diseases.
Fact Sheet